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Paving the way for gene therapy in Huntington's disease

Prof. Matthew Wood and Dr. Samir El Andaloussi, both of DPAG, have been awarded a new £1m grant from the MRC to study gene therapy for Huntington’s Disease.

Huntington’s is a devastating disorder caused by mutations in a single gene, which result in production of a toxic protein. This protein accumulates in the brain, resulting in the progressive degeneration of nerve and muscle cells leading to death, on average within 25 years of diagnosis.

The Oxford researchers hope to employ a type of naturally occurring cell transport method, called exosomes, to deliver gene therapy directly to the brain. They will test their approach in a mouse model of the disease in the hope of proving that this could be a potential mechanism of delivering human therapy.